Testing a new oral medication for advanced cancers

Plain-English translation of NCT04503265 on ClinicalTrials.gov ↗ · Source last updated May 2025 · Translation generated June 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This trial is testing a new medication called AMXI-5001 for people with advanced cancers that have stopped responding to standard treatments or aren't suitable options anymore. The medication works in two ways: it helps repair damage to cancer cells' DNA and interferes with how cancer cells divide. Researchers want to find the right dose and learn how well this treatment works for different types of cancer.

Many people with advanced cancers run out of effective treatment options because their cancer becomes resistant to standard therapies. This medication is being tested because it uses a different approach to attack cancer cells, which may help people whose cancers no longer respond to existing treatments.

Do you qualify?

You likely qualify if…

you have been diagnosed with advanced cancer that has either spread, stopped responding to standard treatment, or for which standard treatments are not suitable for you
your cancer can be measured or seen on scans or through lab tests
you are in good enough overall health to tolerate the study medication (ECOG performance status 0-1)
you are 18 years of age or older
you are able to understand and sign an informed consent form

You likely don't qualify if…

you are currently receiving other cancer treatments at the time you would enroll
you have serious heart disease or abnormal heart rhythm patterns
you have a serious medical condition affecting a major organ system
you are taking certain medications that interact with this treatment (such as some antibiotics, antifungals, or anticonvulsants) or have taken them within the past week
you have been diagnosed with a different cancer (other than your target cancer) in the past two years, or currently have one

What participation looks like

You would take the medication by mouth twice daily throughout the study. The trial is divided into two phases: first, researchers test increasing doses to find the safest and most effective dose, then additional participants join to confirm that dose works and gather more information about how well it controls the cancer. You would visit the study site regularly for blood tests, scans, and checkups to monitor how you are doing and check for any side effects.

AI-generated summary from trial data · Jun 15, 2026 · Not medical advice

Trial locations(4 sites)

United States

University of California, Los Angles (UCLA) Department of Medicine - Hematology/Oncology, Los Angeles, California

Johns Hopkins, Baltimore, Maryland

SCRI Oncology Partners, Nashville, Tennessee

The University of Texas MD Anderson Cancer Center, Houston, Texas